Trial Outcomes & Findings for Ibudilast (MN-166) in Subjects With Amyotrophic Lateral Sclerosis (ALS) (NCT NCT02238626)

Last Updated: 2021-11-05

Results Overview

Safety will be assessed by monitoring and recording all treatment-emergent adverse events (TEAEs) including serious adverse events (SAEs) and discontinuations due to TEAEs and Additional assessments will include regular monitoring of hematology, blood chemistry, and urine values, regular measurement of vital signs, ECGs, medical history, physical and neurological examinations.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

70 participants

Primary outcome timeframe

6 months

Results posted on

2021-11-05

Participant Flow

Participant milestones

Participant milestones
Measure	Placebo (for MN-166) (Early ALS Cohort) Sugar pill manufactured for MN-166 10 mg tablets plus 50 mg riluzole by mouth twice daily for 6 months. Placebo (for MN-166) riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS onset is 5 years or less.	MN-166 (Early ALS Cohort) MN-166 10 mg tablets (up to 60 mg/day) by mouth 2-3 times a day plus 50 mg riluzole 2 times a day by mouth for 6 months. MN-166 riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS onset is 5 years or less.	Placebo (Advanced ALS Cohort) Sugar pill manufactured for MN-166 10 mg tablets plus 50 mg riluzole by mouth twice daily for 6 months. Placebo (for MN-166) riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS onset more than 5 years, but less than 10 years.	MN-166 (Advanced ALS Cohort) MN-166 10 mg tablets (up to 60 mg/day) by mouth 2-3 times a day plus 50 mg riluzole 2 times a day by mouth for 6 months. MN-166 riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS onset is less than 10 years, but more than 5 years.
Overall Study STARTED	17	34	8	11
Overall Study COMPLETED	12	20	5	4
Overall Study NOT COMPLETED	5	14	3	7

Reasons for withdrawal

Reasons for withdrawal
Measure	Placebo (for MN-166) (Early ALS Cohort) Sugar pill manufactured for MN-166 10 mg tablets plus 50 mg riluzole by mouth twice daily for 6 months. Placebo (for MN-166) riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS onset is 5 years or less.	MN-166 (Early ALS Cohort) MN-166 10 mg tablets (up to 60 mg/day) by mouth 2-3 times a day plus 50 mg riluzole 2 times a day by mouth for 6 months. MN-166 riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS onset is 5 years or less.	Placebo (Advanced ALS Cohort) Sugar pill manufactured for MN-166 10 mg tablets plus 50 mg riluzole by mouth twice daily for 6 months. Placebo (for MN-166) riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS onset more than 5 years, but less than 10 years.	MN-166 (Advanced ALS Cohort) MN-166 10 mg tablets (up to 60 mg/day) by mouth 2-3 times a day plus 50 mg riluzole 2 times a day by mouth for 6 months. MN-166 riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS onset is less than 10 years, but more than 5 years.
Overall Study Adverse Event	3	5	0	3
Overall Study Lost to Follow-up	2	4	2	2
Overall Study Withdrawal by Subject	0	3	0	1
Overall Study Death	0	0	1	1
Overall Study Other	0	2	0	0

Baseline Characteristics

Ibudilast (MN-166) in Subjects With Amyotrophic Lateral Sclerosis (ALS)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Placebo (for MN-166) (Early ALS Cohort) n=17 Participants Sugar pill manufactured for MN-166 10 mg tablets plus 50 mg riluzole by mouth twice daily for 6 months. Placebo (for MN-166) riluzole: Patient is given 50 mg riluzole twice daily.	MN-166 (Early ALS Cohort) n=34 Participants MN-166 10 mg tablets (up to 60 mg/day) by mouth 2-3 times a day plus 50 mg riluzole 2 times a day by mouth for 6 months. MN-166 riluzole: Patient is given 50 mg riluzole twice daily.	Placebo (for MN-166) (Advanced ALS Cohort) n=8 Participants Sugar pill manufactured for MN-166 10 mg tablets plus 50 mg riluzole by mouth twice daily for 6 months. Placebo (for MN-166) riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS symptom onset between 5 and 10 years of Screening.	MN-166 (Advanced ALS Cohort) n=11 Participants MN-166 10 mg tablets (up to 60 mg/day) by mouth 2-3 times a day plus 50 mg riluzole 2 times a day by mouth for 6 months. MN-166 riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS symptom onset between 5 and 10 years before screening.	Total n=70 Participants Total of all reporting groups
Age, Continuous	57.2 years STANDARD_DEVIATION 9.8 • n=5 Participants	60.4 years STANDARD_DEVIATION 10.9 • n=7 Participants	56.9 years STANDARD_DEVIATION 7.4 • n=5 Participants	61.6 years STANDARD_DEVIATION 8.1 • n=4 Participants	58.9 years STANDARD_DEVIATION 9.5 • n=21 Participants
Sex: Female, Male Female	5 Participants n=5 Participants	11 Participants n=7 Participants	2 Participants n=5 Participants	5 Participants n=4 Participants	23 Participants n=21 Participants
Sex: Female, Male Male	12 Participants n=5 Participants	23 Participants n=7 Participants	6 Participants n=5 Participants	6 Participants n=4 Participants	47 Participants n=21 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	17 Participants n=5 Participants	34 Participants n=7 Participants	8 Participants n=5 Participants	11 Participants n=4 Participants	70 Participants n=21 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	1 Participants n=21 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) Black or African American	2 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants	0 Participants n=4 Participants	5 Participants n=21 Participants
Race (NIH/OMB) White	15 Participants n=5 Participants	31 Participants n=7 Participants	6 Participants n=5 Participants	11 Participants n=4 Participants	63 Participants n=21 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	1 Participants n=21 Participants
Region of Enrollment United States	17 Participants n=5 Participants	34 Participants n=7 Participants	8 Participants n=5 Participants	11 Participants n=4 Participants	70 Participants n=21 Participants

PRIMARY outcome

Timeframe: 6 months

Population: Safety analysis population included all randomized subjects who received at least one dose of study drug and had at least one post-dose safety assessment.

Outcome measures

Outcome measures
Measure	Placebo (for MN-166) (Early ALS Cohort) n=17 Participants Sugar pill manufactured for MN-166 10 mg tablets plus 50 mg riluzole by mouth twice daily for 6 months. Placebo (for MN-166) riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS onset within 5 years.	MN-166 n=34 Participants MN-166 10 mg tablets (up to 60 mg/day) by mouth 2-3 times a day plus 50 mg riluzole 2 times a day by mouth for 6 months. MN-166 riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS onset within 5 years.	Placebo (for MN-166) (Advanced ALS Cohort) n=8 Participants Sugar pill manufactured for MN-166 10 mg tablets plus 50 mg riluzole by mouth twice daily for 6 months. Placebo (for MN-166) riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS symptom onset between 5 and 10 years before Screening.	MN-166 (Advanced ALS Cohort) n=11 Participants MN-166 10 mg tablets (up to 60 mg/day) by mouth 2-3 times a day plus 50 mg riluzole 2 times a day by mouth for 6 months. MN-166 riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS symptom onset between 5 and 10 years before Screening.
Safety and Tolerability of MN-166 60 mg/d Versus Placebo When Administered With Riluzole in Subjects With ALS	17 Participants	34 Participants	8 Participants	11 Participants

SECONDARY outcome

Timeframe: 6 months

Population: Safety Population

Functional activity as assessed by the Amyotrophic Lateral Sclerosis Functional Rating Scale-revised from baseline visit to Month 6. The best possible score is 48; the worst possible score is 0. Typically, ALS scores decline. In this outcome, the change in score will be a negative value, e.g., -4, -8, etc. The higher negative value indicates greater decline in functional activity (change in score -8 is worse than change in score -4).

Outcome measures

Outcome measures
Measure	Placebo (for MN-166) (Early ALS Cohort) n=17 Participants Sugar pill manufactured for MN-166 10 mg tablets plus 50 mg riluzole by mouth twice daily for 6 months. Placebo (for MN-166) riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS onset within 5 years.	MN-166 n=34 Participants MN-166 10 mg tablets (up to 60 mg/day) by mouth 2-3 times a day plus 50 mg riluzole 2 times a day by mouth for 6 months. MN-166 riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS onset within 5 years.	Placebo (for MN-166) (Advanced ALS Cohort) n=8 Participants Sugar pill manufactured for MN-166 10 mg tablets plus 50 mg riluzole by mouth twice daily for 6 months. Placebo (for MN-166) riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS symptom onset between 5 and 10 years before Screening.	MN-166 (Advanced ALS Cohort) n=11 Participants MN-166 10 mg tablets (up to 60 mg/day) by mouth 2-3 times a day plus 50 mg riluzole 2 times a day by mouth for 6 months. MN-166 riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS symptom onset between 5 and 10 years before Screening.
Mean Change in Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised Total Score From Baseline to Month 6	-3.8 score on a scale Standard Deviation 3.91	-4.5 score on a scale Standard Deviation 5.1	-2.2 score on a scale Standard Deviation 2.56	-4.8 score on a scale Standard Deviation 4.92

SECONDARY outcome

Timeframe: 6 months

Population: Subjects who completed Month 6 (double-blind treatment phase)

Change in respiratory function (breathing capacity) from baseline to Month 6, as measured by slow vital capacity, in which the patient breathes into a spirometer slowly until the lungs are cleared of air. SVC is measured in liters (L). The greater the mean change from baseline to 6 months, the worse the outcome. For example, -10 is worse than -6.

Outcome measures

Outcome measures
Measure	Placebo (for MN-166) (Early ALS Cohort) n=14 Participants Sugar pill manufactured for MN-166 10 mg tablets plus 50 mg riluzole by mouth twice daily for 6 months. Placebo (for MN-166) riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS onset within 5 years.	MN-166 n=32 Participants MN-166 10 mg tablets (up to 60 mg/day) by mouth 2-3 times a day plus 50 mg riluzole 2 times a day by mouth for 6 months. MN-166 riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS onset within 5 years.	Placebo (for MN-166) (Advanced ALS Cohort) n=5 Participants Sugar pill manufactured for MN-166 10 mg tablets plus 50 mg riluzole by mouth twice daily for 6 months. Placebo (for MN-166) riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS symptom onset between 5 and 10 years before Screening.	MN-166 (Advanced ALS Cohort) n=9 Participants MN-166 10 mg tablets (up to 60 mg/day) by mouth 2-3 times a day plus 50 mg riluzole 2 times a day by mouth for 6 months. MN-166 riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS symptom onset between 5 and 10 years before Screening.
Respiratory Function	-6.1 Liters Standard Deviation 9.33	-10.1 Liters Standard Deviation 9.89	-12.2 Liters Standard Deviation 7.07	-11.1 Liters Standard Deviation 18.73

SECONDARY outcome

Timeframe: 6 months

Population: Subjects who completed Month 6 (double-blind treatment phase)

Muscle strength measured by manual muscle testing (MMT) and instrumented hand-held dynamometry. Maximum muscle strength is assessed by measuring the best of 2 handgrips represented as kilograms (kg). The greater the change from baseline to month 6, the worse off the subject is. For example, a change of -0.50 is worse than -0.40.

Outcome measures

Outcome measures
Measure	Placebo (for MN-166) (Early ALS Cohort) n=14 Participants Sugar pill manufactured for MN-166 10 mg tablets plus 50 mg riluzole by mouth twice daily for 6 months. Placebo (for MN-166) riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS onset within 5 years.	MN-166 n=32 Participants MN-166 10 mg tablets (up to 60 mg/day) by mouth 2-3 times a day plus 50 mg riluzole 2 times a day by mouth for 6 months. MN-166 riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS onset within 5 years.	Placebo (for MN-166) (Advanced ALS Cohort) n=5 Participants Sugar pill manufactured for MN-166 10 mg tablets plus 50 mg riluzole by mouth twice daily for 6 months. Placebo (for MN-166) riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS symptom onset between 5 and 10 years before Screening.	MN-166 (Advanced ALS Cohort) n=8 Participants MN-166 10 mg tablets (up to 60 mg/day) by mouth 2-3 times a day plus 50 mg riluzole 2 times a day by mouth for 6 months. MN-166 riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS symptom onset between 5 and 10 years before Screening.
Muscle Strength	-3.9 weight (kg) Standard Deviation 5.37	-4.9 weight (kg) Standard Deviation 5.50	-1.3 weight (kg) Standard Deviation 2.37	-4.4 weight (kg) Standard Deviation 3.63

SECONDARY outcome

Timeframe: 6 months

Population: Full Analysis Set

Non-invasive ventilation (NIV) utilization measured by clinically indicated prescription for NIV intervention and time to clinically indicated prescription for NIV intervention in each group (for early ALS subjects only). This is intended to count the number of subjects who had to go on non-invasive ventilation, as prescribed by the Principal Investigator, during study participation.

Outcome measures

Outcome measures
Measure	Placebo (for MN-166) (Early ALS Cohort) n=16 Participants Sugar pill manufactured for MN-166 10 mg tablets plus 50 mg riluzole by mouth twice daily for 6 months. Placebo (for MN-166) riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS onset within 5 years.	MN-166 n=33 Participants MN-166 10 mg tablets (up to 60 mg/day) by mouth 2-3 times a day plus 50 mg riluzole 2 times a day by mouth for 6 months. MN-166 riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS onset within 5 years.	Placebo (for MN-166) (Advanced ALS Cohort) Sugar pill manufactured for MN-166 10 mg tablets plus 50 mg riluzole by mouth twice daily for 6 months. Placebo (for MN-166) riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS symptom onset between 5 and 10 years before Screening.	MN-166 (Advanced ALS Cohort) MN-166 10 mg tablets (up to 60 mg/day) by mouth 2-3 times a day plus 50 mg riluzole 2 times a day by mouth for 6 months. MN-166 riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS symptom onset between 5 and 10 years before Screening.
Use of Non-invasive Ventilation	6 Participants	10 Participants	—	—

SECONDARY outcome

Timeframe: 6 months

Population: Full analysis set

A patient self-reported questionnaire specifically designed to measure 5 areas of health: physical mobility, activities of daily living and independence, eating and drinking, communication, and emotional functioning. The ALSAQ-5 is brief and easy to complete questionnaire and has undergone rigorous testing for validity, reliability, and sensitivity to change and has been shown to be a robust tool for assessing ALS. The lowest possible score is 0 and the highest possible score is 20. The greater the mean decrease from baseline to Month 6, the group was considered worse off. For example, -2 is worse than -1. The greater the mean increase from baseline to Month 6, the group was considered improved. For example, 2 is better than 1.

Outcome measures

Outcome measures
Measure	Placebo (for MN-166) (Early ALS Cohort) n=14 Participants Sugar pill manufactured for MN-166 10 mg tablets plus 50 mg riluzole by mouth twice daily for 6 months. Placebo (for MN-166) riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS onset within 5 years.	MN-166 n=32 Participants MN-166 10 mg tablets (up to 60 mg/day) by mouth 2-3 times a day plus 50 mg riluzole 2 times a day by mouth for 6 months. MN-166 riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS onset within 5 years.	Placebo (for MN-166) (Advanced ALS Cohort) n=6 Participants Sugar pill manufactured for MN-166 10 mg tablets plus 50 mg riluzole by mouth twice daily for 6 months. Placebo (for MN-166) riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS symptom onset between 5 and 10 years before Screening.	MN-166 (Advanced ALS Cohort) n=9 Participants MN-166 10 mg tablets (up to 60 mg/day) by mouth 2-3 times a day plus 50 mg riluzole 2 times a day by mouth for 6 months. MN-166 riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS symptom onset between 5 and 10 years before Screening.
The Mean Change in Baseline to Month 6 in Quality of Life as Measured by the Amyotrophic Lateral Sclerosis Assessment Questionnaire - 5	2.1 score on a scale Standard Deviation 3.00	1.6 score on a scale Standard Deviation 2.77	-0.70 score on a scale Standard Deviation 2.07	4.8 score on a scale Standard Deviation 3.56

SECONDARY outcome

Timeframe: 6 months

Population: Full analysis set

A scale used to provide a global rating of illness severity, improvement, and response to treatment. It is a 3-item observer rating scale and uses a 7-point rating scale. The scale was rated relative to the previous standard of care visit prior to randomization for entry, i.e., -3 much much much worse, -2 much much worse, - 1 much worse, 0 no change, +1 much better, +2 much much better, +3 much, much, much better. Ratings were provided by the Investigator. The greater the mean decrease from baseline to Month 6, the group was considered worse off. For example, -2 is worse than -1. The greater the mean increase from baseline to Month 6, the group was considered improved. For example, 2 is better than 1.

Outcome measures

Outcome measures
Measure	Placebo (for MN-166) (Early ALS Cohort) n=14 Participants Sugar pill manufactured for MN-166 10 mg tablets plus 50 mg riluzole by mouth twice daily for 6 months. Placebo (for MN-166) riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS onset within 5 years.	MN-166 n=32 Participants MN-166 10 mg tablets (up to 60 mg/day) by mouth 2-3 times a day plus 50 mg riluzole 2 times a day by mouth for 6 months. MN-166 riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS onset within 5 years.	Placebo (for MN-166) (Advanced ALS Cohort) n=6 Participants Sugar pill manufactured for MN-166 10 mg tablets plus 50 mg riluzole by mouth twice daily for 6 months. Placebo (for MN-166) riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS symptom onset between 5 and 10 years before Screening.	MN-166 (Advanced ALS Cohort) n=9 Participants MN-166 10 mg tablets (up to 60 mg/day) by mouth 2-3 times a day plus 50 mg riluzole 2 times a day by mouth for 6 months. MN-166 riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS symptom onset between 5 and 10 years before Screening.
Clinical Global Impression of Change (CGIC)	-1.0 score on a scale Standard Deviation 0.78	-1.4 score on a scale Standard Deviation 0.75	-1.3 score on a scale Standard Deviation 0.52	-1.6 score on a scale Standard Deviation 0.73

Adverse Events

Placebo (for MN-166) (Early ALS Cohort)

Serious events: 1 serious events

Other events: 17 other events

Deaths: 0 deaths

MN-166 (Early ALS Cohort)

Serious events: 5 serious events

Other events: 34 other events

Deaths: 1 deaths

Placebo (for MN-166) (Advanced ALS Cohort)

Serious events: 2 serious events

Other events: 8 other events

Deaths: 1 deaths

MN-166 (Advanced ALS Cohort)

Serious events: 3 serious events

Other events: 11 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	Placebo (for MN-166) (Early ALS Cohort) n=17 participants at risk Sugar pill manufactured for MN-166 10 mg tablets plus 50 mg riluzole by mouth twice daily for 6 months. Placebo (for MN-166) riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS onset within 5 years of screening.	MN-166 (Early ALS Cohort) n=34 participants at risk MN-166 10 mg tablets (up to 60 mg/day) by mouth 2-3 times a day plus 50 mg riluzole 2 times a day by mouth for 6 months. MN-166 riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS onset within 5 years of screening.	Placebo (for MN-166) (Advanced ALS Cohort) n=8 participants at risk Sugar pill manufactured for MN-166 10 mg tablets plus 50 mg riluzole by mouth twice daily for 6 months. Placebo (for MN-166) riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS symptom onset between 5 and 10 years of Screening.	MN-166 (Advanced ALS Cohort) n=11 participants at risk MN-166 10 mg tablets (up to 60 mg/day) by mouth 2-3 times a day plus 50 mg riluzole 2 times a day by mouth for 6 months. MN-166 riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS symptom onset between 5 and 10 years before screening.
Gastrointestinal disorders dysphagia	0.00% 0/17 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	2.9% 1/34 • Number of events 1 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/8 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/11 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).
Gastrointestinal disorders intestinal obstruction	0.00% 0/17 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	2.9% 1/34 • Number of events 1 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/8 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/11 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).
Respiratory, thoracic and mediastinal disorders pneumonia	0.00% 0/17 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	2.9% 1/34 • Number of events 1 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	12.5% 1/8 • Number of events 1 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/11 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).
Respiratory, thoracic and mediastinal disorders pneumonia aspiration	0.00% 0/17 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/34 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/8 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	9.1% 1/11 • Number of events 1 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).
Musculoskeletal and connective tissue disorders Lower limb fracture	0.00% 0/17 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	2.9% 1/34 • Number of events 1 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/8 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/11 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).
Renal and urinary disorders Hydronephrosis	0.00% 0/17 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	2.9% 1/34 • Number of events 1 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/8 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/11 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).
Renal and urinary disorders Ureterolithiasis	0.00% 0/17 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	2.9% 1/34 • Number of events 1 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/8 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/11 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).
Vascular disorders Deep vein thrombosis	0.00% 0/17 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	2.9% 1/34 • Number of events 1 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/8 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/11 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).
Injury, poisoning and procedural complications Ankle fracture	5.9% 1/17 • Number of events 1 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/34 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/8 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/11 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).
Nervous system disorders amyotrophic lateral sclerosis	0.00% 0/17 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/34 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	12.5% 1/8 • Number of events 1 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	9.1% 1/11 • Number of events 1 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).
Respiratory, thoracic and mediastinal disorders aspiration	0.00% 0/17 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/34 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/8 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	9.1% 1/11 • Number of events 1 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).

Other adverse events

Additional Information

Director, Scientific Affairs

MediciNova, Inc.

Phone: 8583444535

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Placebo (for MN-166) (Early ALS Cohort) n=17 participants at risk Sugar pill manufactured for MN-166 10 mg tablets plus 50 mg riluzole by mouth twice daily for 6 months. Placebo (for MN-166) riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS onset within 5 years of screening.	MN-166 (Early ALS Cohort) n=34 participants at risk MN-166 10 mg tablets (up to 60 mg/day) by mouth 2-3 times a day plus 50 mg riluzole 2 times a day by mouth for 6 months. MN-166 riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS onset within 5 years of screening.	Placebo (for MN-166) (Advanced ALS Cohort) n=8 participants at risk Sugar pill manufactured for MN-166 10 mg tablets plus 50 mg riluzole by mouth twice daily for 6 months. Placebo (for MN-166) riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS symptom onset between 5 and 10 years of Screening.	MN-166 (Advanced ALS Cohort) n=11 participants at risk MN-166 10 mg tablets (up to 60 mg/day) by mouth 2-3 times a day plus 50 mg riluzole 2 times a day by mouth for 6 months. MN-166 riluzole: Patient is given 50 mg riluzole twice daily. Patient ALS symptom onset between 5 and 10 years before screening.
Musculoskeletal and connective tissue disorders muscle weakness	52.9% 9/17 • Number of events 9 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	52.9% 18/34 • Number of events 18 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	12.5% 1/8 • Number of events 1 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	36.4% 4/11 • Number of events 4 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).
Injury, poisoning and procedural complications Fall	47.1% 8/17 • Number of events 8 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	41.2% 14/34 • Number of events 14 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	12.5% 1/8 • Number of events 1 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	45.5% 5/11 • Number of events 5 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).
General disorders Fatigue	23.5% 4/17 • Number of events 4 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	35.3% 12/34 • Number of events 12 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	25.0% 2/8 • Number of events 2 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/11 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).
Metabolism and nutrition disorders Decreased appetite	0.00% 0/17 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	26.5% 9/34 • Number of events 9 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/8 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	18.2% 2/11 • Number of events 2 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).
Musculoskeletal and connective tissue disorders muscle spasms	11.8% 2/17 • Number of events 2 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	20.6% 7/34 • Number of events 7 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	25.0% 2/8 • Number of events 2 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	27.3% 3/11 • Number of events 3 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).
Gastrointestinal disorders nausea	11.8% 2/17 • Number of events 2 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	20.6% 7/34 • Number of events 7 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/8 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/11 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).
Gastrointestinal disorders weight decreased	11.8% 2/17 • Number of events 2 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	20.6% 7/34 • Number of events 7 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	25.0% 2/8 • Number of events 2 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	27.3% 3/11 • Number of events 3 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).
Respiratory, thoracic and mediastinal disorders dyspnea	5.9% 1/17 • Number of events 1 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	17.6% 6/34 • Number of events 6 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/8 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/11 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).
Injury, poisoning and procedural complications injury	17.6% 3/17 • Number of events 3 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	14.7% 5/34 • Number of events 5 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/8 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/11 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).
Nervous system disorders headache	17.6% 3/17 • Number of events 3 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	11.8% 4/34 • Number of events 4 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/8 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/11 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).
Musculoskeletal and connective tissue disorders mobility decreased	17.6% 3/17 • Number of events 3 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/34 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/8 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/11 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).
Gastrointestinal disorders dysphagia	29.4% 5/17 • Number of events 5 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	8.8% 3/34 • Number of events 3 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/8 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/11 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).
Gastrointestinal disorders salivary hypersecretion	0.00% 0/17 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/34 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	25.0% 2/8 • Number of events 2 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	27.3% 3/11 • Number of events 3 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).
Skin and subcutaneous tissue disorders skin abrasion	0.00% 0/17 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/34 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	12.5% 1/8 • Number of events 1 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	27.3% 3/11 • Number of events 3 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).
Respiratory, thoracic and mediastinal disorders respiratory distress	0.00% 0/17 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/34 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/8 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	27.3% 3/11 • Number of events 3 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).
Gastrointestinal disorders abdominal distention	0.00% 0/17 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/34 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	25.0% 2/8 • Number of events 2 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	18.2% 2/11 • Number of events 2 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).
Gastrointestinal disorders diarrhea	0.00% 0/17 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/34 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	25.0% 2/8 • Number of events 2 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	18.2% 2/11 • Number of events 2 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).
Respiratory, thoracic and mediastinal disorders upper respiratory tract infection	0.00% 0/17 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/34 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	25.0% 2/8 • Number of events 2 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	18.2% 2/11 • Number of events 2 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).
Gastrointestinal disorders gastrostomy	0.00% 0/17 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/34 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	12.5% 1/8 • Number of events 1 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	18.2% 2/11 • Number of events 2 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).
Vascular disorders edema peripheral	0.00% 0/17 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/34 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	37.5% 3/8 • Number of events 3 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).	0.00% 0/11 • Adverse events were collected from Baseline to end of double-blind treatment (6 months).